OUR DRUG DISCOVERY PIPELINE

Propionic acidemia (PA) and methylmalonic acidemia (MMA) are inborn errors of metabolism in the branched-chain amino acid catabolism pathways, characterized by the build-up of toxic organic acids that result in pathophysiologic effects leading to significant morbidity and mortality in afflicted children. The prevalence of PA and MMA in the US and Europe is estimated to be ~2000 and ~4000, respectively. Children afflicted with these diseases are identified through newborn screening, yet very little is available in terms of treatment other than an extremely restrictive diet. Unfortunately, life expectancy is brief with nearly all patients dying in the first quartile of life.

HemoShear Therapeutics is developing treatments for PA and MMA. We have recapitulated the biology of these diseases using tissue from patients and our REVEAL-Tx™ platform, allowing us to interrogate the disease pathways, and select and test meaningful drug targets to combat the diseases. Chemistry programs were initiated against our targets in 2016 and clinical trials are expected to start enrolling in late 2018.

Nonalcoholic steatohepatitis (NASH) is a serious, chronic liver disease that is estimated to impact over 16 million people in the United States alone. NASH is characterized by inflammation and excessive fat accumulation in the liver that progresses to fibrosis, cirrhosis, liver cancer and eventually liver failure. There is currently no FDA approved therapeutic available for NASH and liver fibrosis.